Raleigh-based Innovate Biopharmaceuticals has received U.S. Food and Drug Administration orphan drug designation for an experimental oral drug to treat pediatric ulcerative colitis.

The status could help expedite the therapy’s development and approval.

Under the Orphan Drug Act of 1983, the FDA provides incentives for companies developing treatments that are expected to provide significant therapeutic advantage over existing treatments and that target rare medical conditions affecting fewer than 200,000 U.S. patients per year. Incentives include seven-year market exclusivity for an approved orphan drug, tax credits on U.S. clinical trials, fast-tracking of regulatory proceedings, and exemption from certain fees.

Innovate’s orphan drug, called INN-108, is a novel small molecule that combines two agents:  mesalamine, or 5-aminosalicylic acid, an approved agent for ulcerative colitis; and 4-aminophenylacetic acid, an immunomodulatory agent approved in Japan for rheumatoid arthritis. The two agents are chemically bonded but are separated enzymatically when they reach the colon, or large intestine.

Innovate reported that preliminary data show the combination therapy could be a more effective drug than approved mesalamine/5-ASA treatments alone.

INN-108 has successfully completed two Phase 1 clinical trials for mild to moderate ulcerative colitis in both healthy adult subjects and adults with ulcerative colitis. It will enter a Phase 2 trial in 2018.

A liquid oral formulation of INN-108 for ulcerative colitis is in development for greater convenience in pediatric use.

Ulcerative colitis is a chronic inflammatory bowel disease that mainly affects the colon. Symptoms typically include constant diarrhea mixed with blood, abdominal pain, increased bowel movements and, in severe cases, weight loss and fatigue. Children can also develop unique complications related to growth, development, nutrition, pubertal maturation, bone mineral density accretion and psychological impacts.

Though the disease can present at any age, it often begins in teenagers and young adults. Various studies estimate that there are between 13,700 and 87,600 cases of pediatric ulcerative colitis in the United States, with most of those occurring in children aged 10 to 17.

Celiac drug nears Phase 3 trials

Innovate is developing another drug candidate, larazotide acetate (INN-202), for the potential treatment of celiac disease, a type of inflammatory bowel disease that affects 3 million people in the United States and about 15 million worldwide.

Larazotide, an oral peptide formulated into a capsule, decreases intestinal permeability – also known as “leaky gut” – and regulates tight junctions by reducing antigen trafficking across epithelial cells in the intestines.

Leaky gut has been widely recognized in the scientific literature as a gateway to multiple autoimmune diseases, including celiac disease, irritable bowel syndrome, inflammatory bowel diseases such as Crohn’s and ulcerative colitis, type 1 diabetes mellitus, nonalcoholic steatohepatitis, chronic kidney disease and several others.

Larazotide has successfully met its primary endpoint in an efficacy clinical trial for celiac disease, and Phase 3 clinical trials are expected to begin later this year, the company reported. Larazotide has received Fast Track designation from the FDA.

Reverse merger

Innovate is a clinical stage biotechnology company focused on developing novel medicines for autoimmune and inflammatory disorders. The company announced in July it had signed a definitive merger agreement with California-based Monster Digital under which the shareholders of privately held Innovate will become the majority owners of Monster Digital, subject to shareholder approval.

The transaction is expected to close in 2017, and the combined company will trade on the Nasdaq Capital Market stock exchange under a new ticker symbol.

The merger will give Innovate access to the public markets faster than a traditional initial public offering, helping it expedite drug development and expand its product pipeline, the company said when announcing the merger agreement.

(C) N.C. Biotechnology Center